NICE Still Opposes Adding Fampyra to NHS for England
The National Institute for Health and Care Excellence (NICE) in England is set to again recommend against adding Fampyra (fampridine) to the list of medications available to multiple sclerosis (MS) patients with walking difficulties through the country’s national health service (NHS).
The poor cost-effectiveness of Fampyra — sold as Ampyra in the U.S. and with generics also available — has been cited as NICE’s persistent concern (since 2014) in its draft guidance for the management of MS.
While this was also the reason behind repeated rejections by the Scottish Medicines Consortium, that agency favored Fampyra’s inclusion in April 2020 following a confidential discount offered by Biogen, which markets the therapy in Europe, that improved its cost-effectiveness.
Fampyra has also been available at no cost to patients living in Wales since December 2019 as part of that country’s patient access scheme — an arrangement where the medicine is offered on an exceptional basis through an agreement with the pharmaceutical company. This type of agreement is usually set for a particular period of time while more data are collected to help address the uncertainties identified during an initial evaluation.
Based on these disparities in access, MS organizations have been calling for an end to the so-called “postcode lottery,” emphasizing that 40,000 MS patients in England and Northern Ireland are denied lower- or no-cost access to Fampyra. They may acquire the oral therapy through private prescriptions at a reported monthly cost of between £200 and £600 (about $264 to $792).
Patients, caregivers, healthcare professionals, and advocates now have the opportunity to comment on NICE’s draft guidance. A consultation period will run until 5 p.m. on Jan. 31, after which the appraisal committee will consider the evidence and comments before making a final recommendation, expected on June 8.
“It’s deeply disappointing that NICE has decided not to recommend [Fampyra] for use on the NHS in England,” Phillip Anderson, the head of policy and evidence for the U.K.’s MS Society, said in a press release.
“Living with MS is relentless, painful and disabling — but we know that [Fampyra] can make a significant difference to people’s walking ability, helping them to live more independent and active lives,” Anderson added.
“NICE, NHS England and the manufacturer, Biogen, need to find a solution to make sure everyone who’s eligible for fampridine can access it,” Anderson said.
Fampyra/Ampyra, developed by Acorda Therapeutics, is an extended-release oral tablet that aids walking in MS patients by improving electric signal conduction through nerve fibers in the brain and spinal cord.
The therapy was approved in the U.S. in 2010 to improve walking abilities in MS patients. It was granted a conditional approval in Europe for the same indication in 2011, followed by a full approval in 2017. It is available as a 10 mg tablet, taken without food twice a day, in the morning and at night.
Regulatory approvals were based on data from two Phase 3 clinical trials, showing that a significantly greater proportion of patients receiving the therapy had improvements in walking speed compared with those given a placebo (34.8–42.9% vs. 8.3–9.3%).
Fampyra “was shown to be effective in treating lack of mobility in some people with MS, but not all,” NICE stated in its draft.
A subsequent meta-analysis of nine placebo-controlled trials showed that the oral therapy aids not only patients’ walking abilities but also their finger dexterity and cognitive function.
The therapy, however, carries a known risk of adverse reactions that include seizures and allergic shock, which increases with dose exposure.